Heart devices often approved without comparisons

NEW YORK (Reuters Health) - Many new heart devices, such as valves and stents, are approved for use by the U.S. Food and Drug Administration (FDA) without good studies showing that they offer any benefits beyond existing treatments, according to a new study.

"This really leaves open the question of, 'are you better off with this new device or whatever conventional therapy is already?'" said Dr. Rita Redberg, one of the authors of the study and a professor at the University of California, San Francisco.

Given that devices often require surgery, "from a patient point of view, you're taking a lot more risk with a device, so the harms are potentially much greater," added Redberg, whose team published the work in the Journal of the American Medical Association. "So it's really very important to know that the device is an improvement over what you could have had."

High-risk heart devices, which include implanted defibrillators, mechanical pumps and tubes called stents, go through an approval process called "premarket approval," which includes a more stringent review of the evidence than for less risky devices.

However, the requirements for the approval of devices in general are less strict than for drugs.

The FDA requires two studies comparing the drug to a "control" - typically, the current standard of care or a fake pill called a placebo - before it will approve a drug.

For first-of-a-kind devices, FDA does require controlled trials, FDA spokeswoman Michelle Bolek told Reuters Health by email. However, the agency does not have such demands for devices that aren't first-of-a-kind, and approval can be given without direct comparisons to controls, depending on the use of the device and the extent of experience doctors have had with it.

The FDA said there are good reasons why comparing new devices to other treatments or older generations of a product might not be feasible or ethical.

"And, requiring them could significantly and unnecessarily delay patient access to critical new technology," Bolek added.

COMPARATIVE EFFECTIVENESS RESEARCH

Recent studies have found that sometimes patients are given newer and more expensive products without a clear indication that they're better off for it (see Reuters report of February 28, 2012 and Reuters Health report of July 9, 2012).

The 2010 Patient Protection and Affordable Care Act, commonly called Obamacare, established the Patient-Centered Outcomes Research Institute, which funds research on comparing the effectiveness of different approaches to medical care.

To see how often heart devices are compared to existing therapies as part of the approval process, Redberg and her colleagues collected information from the FDA on all high-risk device approvals from 2000 to 2011.

They found that 40 percent of approved devices had been in studies comparing them to other treatments.

The comparison treatment could be an older generation of the same implant, a medication or a surgical procedure.

The rest of the approved devices relied on either the results from other studies in which controls were used, but the new device was not compared head-to-head with other treatments; from expectations of how well the device should work; or from research with no comparisons.

"We were surprised and disappointed," said Redberg, who is also editor of the Archives of Internal Medicine and a member of the FDA's Drug Administration Circulatory System Devices Panel. "We had already seen (in a previous study) there wasn't as many randomized controlled trials as one would hope for devices, but we didn't appreciate it until we looked at the data."

SOMETIMES JUSTIFIABLE?

Some devices were less likely to have been compared to a conventional therapy than others.

Ventricular assist devices, for instance, used in severe cases of heart failure, were approved without any comparison to another therapy, but this could be justified, said Dr. David Brown, a professor at Stony Brook University in New York, who was not involved in the study.

"People are near death when they need the device, and it may not be ethical or practical to design a trial that compares it to something else," he said.

"However, if you look at most of the devices in the other categories, they're not anywhere near being performed in people who don't have other options," Brown told Reuters Health.

For instance, there are alternatives to new cardiac stents, pacemakers and defibrillators, such as drugs or other versions of the same devices, he pointed out.

Yet only about four out of 10 approvals for these types of devices included studies that compared them to existing treatments.

The FDA's Bolek said the information Redberg's group used to evaluate the studies conducted to get a device approved "does not reflect all of the data reviewed by the FDA in a (premarket approval) submission."

Bolek said FDA weighs the quality of studies and their results, tests performed outside of the clinic and data monitoring committees.

Congress, Bolek said, has authorized the FDA to take the "least burdensome provision" to approve devices, to ensure that patients are not denied early access to new medical technology.

Dr. Ron Waksman, the associate director of the division of cardiology at the MedStar Washington Hospital Center, said the study does not differentiate between new products and slight improvements on already-approved devices.

"You don't need to...for every change and iteration, do a clinical randomized trial" said Waksman, who was not part of the study but has been a consultant to medical device companies.

Still, Dr. William Boden at the Albany VA Stratton Medical Center said the different requirements for drug and device approvals is "egregious."

"I think (the study) really elucidates the fact that there is just an unfortunate double standard, where there is a lack of transparency and a critical need for more comparative effectiveness evaluation before these devices come to market," said Boden, who did not participate in the current research but whose research has been funded by pharmaceutical companies.

Brown said consumers should express their concerns about a lack of comparative effectiveness studies to their representatives in Congress.